SRK-015 for Spinal Muscular Atrophy (TOPAZ Trial)

"SRK-015 has been studied since 2019, beginning with an inaugural trial sponsored by Scholar Rock Inc. which involved 58 participants. This drug subsequently received its Phase 2 approval in the same year and is currently being evaluated in two active studies conducted across 24 cities and 9 nations worldwide."

"This research project is no longer recruiting participants. It first appeared on April 22nd 2019 and the latest update was October 24th 2022. If you are seeking other trials, there are 233 studies actively looking for patients with muscular atrophy as well as 2 clinical assessments that need guinea pigs to investigate SRK-015."

"This medical trial has open enrolment in 11 different locations, including Norfolk, New york and Phoenix. To reduce travel time if you choose to partake, it is wise to select the closest site from your location."

"At this time, enrollment for the clinical trial has been closed. Initially posted in April 2019 and last updated October 2022, no more participants are being accepted at present. However, 233 other studies related to muscular atrophy are still actively recruiting volunteers and two trials for SRK-015 as well."

"The youngest participants eligible for the trial must be two years old and no older than twenty-one at the time of enrollment."

"Preliminary clinical evidence suggests SRK-015 is safe, thus it has been given a score of 2 on the safety scale. As this is still in Phase 2 trials though, more data needs to be collected before efficacy can be determined."

"Currently, two studies are in progress investigating SRK-015. One of them is in its final phase (Phase 3). While the main research site for these trials is San Diego, California, there are an additional 53 sites conducting related investigations."

"This trial, to be assessed over a Baseline up to 12 months period, aims to evaluate the Change From Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) Total Score at Day 364 (Visit 15). It also considers the secondary outcomes of Cohort 1: Change From Baseline in Timed Rise From Floor (From RHS), Cohort 2 &3: Change From Baseline in HFMSE Total Score at Other Prespecified Timepoints and Cohort 1's Change From Baseline in 10-Meter Walk/Run."

"This clinical trial requires 58 participants, aged between two and 21 years old, who have been diagnosed with muscular atrophy. Additionally, they must be able to satisfy the following criteria: Non-ambulatory individuals will need to demonstrate their ability to remain upright for 10 seconds without external support; patients that are eligible should possess a later-onset SMA diagnosis prior to receiving any treatment; those enrolled in this study should expect IV infusions and blood sample collection over an extended period of time; nutritional status must not have changed significantly in the six months leading up ambulating patients must be capable of traversing ten meters within 30 seconds (for cohorts"